A revolutionary Melbourne-led project may soon redefine the global fight against Alzheimer’s disease, with scientists developing a world-first therapy that could prevent—or even repair—the neurological damage caused by the condition.
The treatment, which will be delivered as a nasal spray, is expected to enter clinical trials in Melbourne within the next two years.
The initiative is being spearheaded by internationally recognised IVF and stem-cell pioneer Professor Alan Trounson, who today launches a new Melbourne-based biotechnology company, Evinco Therapeutics, dedicated to bringing the therapy from the laboratory to human testing.
Professor Trounson said the therapy represents “a new frontier in the fight against Alzheimer’s disease and other neuro-inflammatory conditions,” noting that no other research group is currently taking such a direct immune-repair approach.
The treatment works by using extracellular vesicles (EVs) harvested from natural killer (NK) cells, the body’s first immune responders. Although NK cells are highly effective in targeting unwanted or diseased cells, they cannot cross the blood–brain barrier. Their EVs, however, can. These tiny structures can pass directly into the brain and instruct microglia—the brain’s own immune cells—to degrade toxic amyloid protein aggregates and reduce inflammation, two hallmarks of Alzheimer’s disease.
Evinco Therapeutics will begin safety studies in rodents next year, building on years of innovation including the development of large NK-cell banks at Cartherics, Professor Trounson’s previous company. His team has successfully extracted and purified EVs from NK cells, showing their ability to activate microglia and influence both amyloid plaques and tau proteins linked to Alzheimer’s progression.
The idea for this approach emerged after Trounson learned of a Korean group that treated cancer and chemotherapy patients with their own NK cells in the United States. By chance, they administered the therapy to an Alzheimer’s patient—who suddenly began speaking again after two years of silence. Subsequent patients displayed similar improvements. In a small FDA phase II trial, 12 of 13 Alzheimer’s patients showed notable clinical responses.
However, as Trounson notes, most NK cells injected into the bloodstream remain outside the brain, limiting their effect. This led his team to focus on EVs as a more direct and efficient alternative, delivered nasally to bypass the blood–brain barrier entirely.
If successful, the therapy may also have applications beyond Alzheimer’s, including Parkinson’s disease, traumatic brain injuries, concussion, and fibromyalgia.
A major pharmaceutical company has already partnered with Evinco Therapeutics to design the upcoming proof-of-concept studies. While researchers cannot yet confirm whether the therapy can reverse Alzheimer’s, they are optimistic about its potential to prevent progression and reduce symptoms.
The EV-based nasal spray is inexpensive to produce, freeze-dryable, and easily administered—opening the door to widespread accessibility should trials confirm its effectiveness.
“I’m really excited because I cannot see any barriers to finding out whether this works,” Professor Trounson said. “And we may know within two years.”
